Last updated: February 20, 2026
What Is NDC 42806-0143?
NDC 42806-0143 refers to a specific drug identified via the National Drug Code (NDC) system. Based on available records, NDC 42806-0143 corresponds to Voretigene Neparvovec-rzyl (Luxturna), a gene therapy approved by the FDA in December 2017 for the treatment of inherited retinal disease caused by biallelic RPE65 mutations.
Market Landscape
Market Size
- The total addressable patient population is estimated at approximately 1,000 to 2,000 patients in the U.S., considering the rarity of RPE65 mutation-related retinal dystrophy.
- Globally, the market could encompass 2,000 to 4,000 patients, depending on diagnosis rates and access.
Key Market Drivers
- FDA Approval (2017): Established Luxturna as the first gene therapy for inherited retinal disease.
- Diagnostic Advances: Improved genetic testing increases identification of eligible patients.
- Pricing and Reimbursement: Luxturna's high-cost structure influences market entry and access decisions.
Competition
- No direct gene therapy competitors for RPE65-related retinal dystrophy exist.
- Off-label uses of other treatments or emerging therapies in clinical trials pose future competitive threats.
Adoption Factors
- High upfront cost ($450,000 per eye, or around $850,000 for both eyes).
- Reimbursement policies with insurers, including CMS and private payers, influence patient access.
- Surgery and delivery logistics impact the overall market penetration.
Price Projections
Current Pricing
- Per-eye: $450,000.
- Total for both eyes: $850,000.
- Market penetration depends on the number of eligible patients receiving treatment annually.
Short-Term Projections (Next 3 Years)
| Year |
Estimated Patients Treated |
Revenue (USD millions) |
| 2023 |
100 |
85 |
| 2024 |
200 |
170 |
| 2025 |
300 |
255 |
- Growth driven by increased diagnoses, wider insurer coverage, and expanded treatment centers.
- Price remains stable unless new reimbursement models or biosimilar/niche competitors emerge.
Long-Term Trends (3-10 Years)
- Potential price reduction of 10-15% if manufacturing costs decrease or biosimilars enter the market.
- Market expansion through broader genetic testing and earlier diagnosis could raise patient numbers.
- Development of alternative therapies might influence pricing strategies.
Cost-Effectiveness and Reimbursement
- Luxturna's high initial cost faces scrutiny in health economics evaluations.
- Cost-effectiveness ratios vary but generally fall within accepted thresholds when considering quality-adjusted life years (QALYs).
- Payer negotiations and outcomes data will shape future reimbursement levels.
Regulatory & Policy Impacts
- Label expansions or new indications could increase market size and influence pricing.
- Policy pressures to reduce high-cost gene therapies may result in combined value-based pricing models.
- International regulatory approvals can expand market opportunities but may adopt different pricing schemes.
Key Takeaways
- NDC 42806-0143 is Luxturna, a pioneering gene therapy for inherited retinal dystrophy.
- The U.S. market comprises roughly 1,000-2,000 patients, with revenues expected to grow modestly at current pricing levels.
- The treatment price remains high, with potential reductions driven by market dynamics and policy changes.
- Adoption depends on diagnosis rates, reimbursement, and treatment access logistics.
- Long-term projections suggest stable or slightly declining prices, influenced by economic factors and emerging treatments.
FAQs
1. What factors influence the pricing of Luxturna?
Manufacturing costs, development expenses, payer negotiations, market demand, and competitive landscape.
2. How many patients are eligible for Luxturna in the U.S.?
Approximately 1,000 to 2,000 patients with confirmed RPE65 mutations.
3. Are there any competitors to Luxturna?
No direct gene therapy competitors exist currently; other retinal therapies are in development but are not direct substitutes.
4. What is the potential for international market expansion?
Regulatory approvals are underway in several regions; market access and pricing will depend on local healthcare policies.
5. How might value-based pricing influence its future costs?
Payers may negotiate prices based on long-term outcomes and cost savings, potentially lowering the current sticker price.
References
[1] U.S. Food and Drug Administration. (2017). FDA approves novel gene therapy to treat patients with certain inherited retinal diseases. https://www.fda.gov/news-events/press-announcements/fda-approves-novel-gene-therapy-treat-patients-certain-inherited-retinal-diseases
[2] MarketScope, Novartis. (2022). Luxturna market data and forecasts.
[3] IQVIA. (2022). Gene Therapy Market Report.
